Alpha-1 antitrypsin deficiency (AATD) is a genetic condition characterised by low circulating levels of alpha-1 antitrypsin (AAT), a serine proteinase inhibitor. The most common deficiency variants are the S and Z mutations, which cause the accumulation of misfolded AAT in hepatocytes resulting in endoplasmic reticular stress and insufficient release of AAT into the circulation (<11μmol/L). This leads to liver disease, as well as an increased risk of emphysema due to unopposed proteolytic activity of neutrophil-derived serine proteinases in the lungs. AATD has been traditionally viewed as an inflammatory disorder caused directly by a proteinase-antiproteinase imbalance in the lung, but increasing evidence suggests that low AAT levels may affect other cellular functions. Recently, AAT polymers have been identified in both monocytes and macrophages from AATD patients and evidence is building that these cells may also play a role in the development of AATD lung disease. Alveolar macrophages are phagocytic cells that are important in the lung immune response but are also implicated in driving inflammation. This review explores the potential implications of monocyte and macrophage involvement in non-liver AAT synthesis and the pathophysiology of AATD lung disease.
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http://dx.doi.org/10.2147/COPD.S276792 | DOI Listing |
Rev Physiol Biochem Pharmacol
January 2025
Institute of Medical Sciences, University of Aberdeen, Aberdeen, Scotland, UK.
Ribosomes use multiple electrical forces to regulate new protein construction, to ensure efficient protein cotranslation, chaperoning, and folding. When these electrical regulatory forces are disrupted as in point charge mutations, specific disease occurs from aberrantly folded proteins. α1 antitrypsin deficiency is perhaps the best-known misfolded protein disease and is covered in some detail.
View Article and Find Full Text PDFBiomedica
December 2024
Departamento de Medicina Interna, Facultad de Salud, Universidad del Valle, Cali, Colombia.
Introduction: Non-cystic fibrosis bronchiectasis is a complex medical condition with multiple etiologies, characterized by chronic productive cough and radiologic evidence of airway lumen dilation and wall thickening. Associated exacerbations and declining lung function contribute to increasing disability and mortality. There are no data about the prevalence of non-cystic fibrosis bronchiectasis etiologies in the Colombian population.
View Article and Find Full Text PDFRev Esp Enferm Dig
January 2025
Hepatology. Gastroenterology Unit, Hospital Universitario Central de Asturias.
A 16-year-old boy was diagnosed from multiple sclerosis (MS) after suffering from paresthesia in upper limbs and VI cranial nerve paresis. Corticosteroids and fingolimod were started. After 13 months a worsening of liver biochemical tests (LBT) was noticed: ALP 787 U/L, GGT 737 U/L, AST 195, ALT 321, Bi 0.
View Article and Find Full Text PDFPatient Relat Outcome Meas
January 2025
Institute of Applied Health Sciences, University of Birmingham, Birmingham, UK.
Alpha-1 antitrypsin deficiency (AATD) is a rare cause of chronic lung and liver disease without its own patient reported-outcome measure (PROM). PROMs for Chronic Obstructive Pulmonary Disease (COPD) are commonly used instead, but AATD differs from COPD in several ways. We reviewed whether the PROMs used in the AATD literature adequately assess quality-of-life in these patients.
View Article and Find Full Text PDFERJ Open Res
January 2025
Kamada Ltd., Rehovot, Israel.
Background: Alpha-1 antitrypsin (AAT)-deficient individuals have a greater risk for developing COPD than individuals with normal AAT levels.
Methods: This was a double-blind, randomised, parallel group, placebo-controlled trial to examine the safety and tolerability of "Kamada-AAT for Inhalation" (inhaled AAT) in subjects with AAT deficiency, and to explore its effect on AAT and biomarkers in the lung epithelial lining fluid (ELF). 36 patients with severe AAT deficiency were randomised 2:1 to receive 80 mg or 160 mg inhaled AAT or placebo once daily for 12 weeks.
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